CER-001 Infusions in Subjects with Familial HDL-c Deficiency

CER-001 Infusions in Subjects with Familial HDL-c Deficiency - PROT- CER-001-CLIN-007

Status

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-006188-23-NL

Enrollment

Registered

2011-12-27

Start date

2012-02-14

Completion date

Unknown

Last updated

2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial HDL-c Deficiency MedDRA version: 14.1 Level: LLT Classification code 10020604 Term: Hypercholesterolemia System Organ Class: 10027433 - Metabolism and nutrition disorders

Interventions

Product Name: Recombinant Human Apolipoprotein A-I/Phospholipids Complex Product Code: CER-001 Pharmaceutical Form: Sterile concentrate Current Sponsor code: CER-001 Other descriptive name: recombinan

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Male or female subjects with genetically confirmed homozygous familial HDL-c deficiency. • Females of childbearing potential that agree and commit to use an acceptable form of birth control for the entire study. Acceptable forms of birth control for this study are defined as a barrier method plus hormonal therapy (implants, injections, oral contraceptives and IUDs) or abstinence. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: • Use of an investigational agent within 30 days of the first dose of CER-001. • Females who are pregnant, breastfeeding, or plan to become pregnant during the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: A pilot study to investigate the safety and efficacy of CER-001 infusions in patients with familial HDL-c deficiency due to defects in genes coding for ApoA-I, ABCA1 or LCAT.;Secondary Objective: • markers of vascular endpoints o vessel wall inflammation (assessed by PET-CT) and o atherosclerosis (assessed by 3T-carotid MRI) • ApoA-I levels (pharmacokinetic parameters) • lipids and lipoproteins (pharmacodynamics parameters) • cholesterol flux • dermatologic and ocular features of HDL deficiency ;Primary end point(s): • Percent change in carotid total vessel area (TVA) and normalized wall index (NWI) assessed by MRI from baseline to Week 26 • Percent change in carotid target to background ratio (TBR) assessed by PET-CT from baseline to Week 4 ;Timepoint(s) of evaluation of this end point: Baseline day 1, week 4, week 26.	—

Measure

Time frame

Main Objective: A pilot study to investigate the safety and efficacy of CER-001 infusions in patients with familial HDL-c deficiency due to defects in genes coding for ApoA-I, ABCA1 or LCAT.;Secondary Objective: • markers of vascular endpoints o vessel wall inflammation (assessed by PET-CT) and o atherosclerosis (assessed by 3T-carotid MRI) • ApoA-I levels (pharmacokinetic parameters) • lipids and lipoproteins (pharmacodynamics parameters) • cholesterol flux • dermatologic and ocular features of HDL deficiency ;Primary end point(s): • Percent change in carotid total vessel area (TVA) and normalized wall index (NWI) assessed by MRI from baseline to Week 26 • Percent change in carotid target to background ratio (TBR) assessed by PET-CT from baseline to Week 4 ;Timepoint(s) of evaluation of this end point: Baseline day 1, week 4, week 26.

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Secondary

Measure	Time frame
Secondary end point(s): Secondary Efficacy Measurements: • Percent change in carotid TVA and NWI assessed by MRI from baseline to Week 4 • Changes in pharmacodynamic parameters over time; • Change in cholesterol flux (TICE and TCE) from baseline to Week 26; and • Changes in dermatologic and ocular features from baseline to Week 4 Pharmacokinetic Measurements: • Single dose pharmacokinetic parameters following the first and final doses Safety and Tolerance: • Adverse event profile • Changes in clinical laboratory measurements • Development of antibodies to ApoA-I and their neutralizing potential;Timepoint(s) of evaluation of this end point: Baseline day 1, week 4, week 26.	—

Measure

Time frame

Secondary end point(s): Secondary Efficacy Measurements: • Percent change in carotid TVA and NWI assessed by MRI from baseline to Week 4 • Changes in pharmacodynamic parameters over time; • Change in cholesterol flux (TICE and TCE) from baseline to Week 26; and • Changes in dermatologic and ocular features from baseline to Week 4 Pharmacokinetic Measurements: • Single dose pharmacokinetic parameters following the first and final doses Safety and Tolerance: • Adverse event profile • Changes in clinical laboratory measurements • Development of antibodies to ApoA-I and their neutralizing potential;Timepoint(s) of evaluation of this end point: Baseline day 1, week 4, week 26.

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Countries

Netherlands

Contacts

Public ContactChristopher Prue

Cerenis Therapeutics

prue@cerenis.com+33(0)674 91 55 36

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026